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Looking ahead, 2025 could represent a major turning point for the pharmaceutical sector. These twins simulate how a patients condition might evolve without treatment, enabling researchers to compare the real-world effects of an experimental therapy against predicted outcomes.
WEDNESDAY, March 19, 2025 -- A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death linked to the treatment. The company behind the drug, Sarepta Therapeutics, said that the.
MONDAY, Jan 6, 2025 -- Turns out, starting physical therapy earlier after a concussion may significantly improve recovery outcomes.A new study published recently in the Physical Therapy & Rehabilitation Journal followed just over 200 adults.
WEDNESDAY, March 19, 2025 -- A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death linked to the treatment.The company behind the drug, Sarepta Therapeutics, said that the patient.
2, 2025 -- Eczema can be maddening, causing thick, scaly patches of dry skin that itch like the devil.Theres no cure for the skin condition, but a new study shows that self-guided talk therapy can help a person deal with the i. THURSDAY, Jan.
MONDAY, March 10, 2025 -- Hormone replacement therapy during menopause appears to be linked to a toxic brain protein thats a hallmark of Alzheimers disease.Women over 70 had a faster accumulation of tau in their brains if theyd taken hormone ther.
TUESDAY, March 18, 2025 -- Gender-affirming hormone therapy may not only help transgender and nonbinary individuals physically -- it could also improve their mental health, new research suggests.A study published March 17 in JAMA Network Open found.
MONDAY, March 10, 2025 -- LED light therapy is touted by social media influencers as a trendy way to treat acne.And it actually appears to work, a new evidence review suggests.Both red and blue LED light therapy are safe and effective in clearing.
THURSDAY, March 20, 2025 -- Doctors should prescribe triptans for migraine patients who arent receiving relief from over-the-counter (OTC) pain relievers, according to a new clinical guideline from the American College of Physicians.The r.
Dr Shalini Andersson, Vice President Nucleic Acid TherapeuticsRNA Gene Cell Therapy at Discovery Sciences, AstraZeneca Dr Shalini Andersson, Vice President Nucleic Acid Therapeutics, AstraZeneca, said: Im honoured to be invited to present at Therapeutic Oligonucleotides 2025.
16, 2025 -- Cutting-edge targeted therapies are pushing back the line between life and death for cancer patients.However, these targeted cancer drugs frequently arent benefitting members of ethnic and racial minorities in the U.S., THURSDAY, Jan.
MONDAY, March 31, 2025 -- Breast-conserving therapy for breast cancer might not conserve as much as previously thought, a new study suggests.Womens breasts can shrink considerably after theyve undergone radiation therapy and lumpectomy for their e.
By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Theres still a long way to go to make this a therapy, Minikel said. Online January 14, 2025. Nature Medicine.
WEDNESDAY, March 5, 2025 -- Sight-robbing injuries to the cornea can be repaired using a groundbreaking experimental stem cell treatment, a new study shows.The cornea -- the clear outermost layer of the eye -- can become irreversibly damaged if.
21, 2025 -- Hormone replacement therapy to ease menopause symptoms doesnt increase a womans risk of brain tumors.Researchers found no link between hormone therapy and gliomas, the most common brain tumors in adults, according to res. TUESDAY, Jan.
2025 [link] Uncovering a drugs mechanism of action and possible adverse effects are critical components in drug discovery and development. Some of this potentially contributes to the adverse effects experienced by patients undergoing therapy using these drugs. Darragh, Andrew M. Hanna, Justin H. Lipner, Alastair J. King, Nicole B.
An ancient RNA-guided system could simplify delivery of gene editing therapies By Corie Lok February 27, 2025 Breadcrumb Home An ancient RNA-guided system could simplify delivery of gene editing therapies The programmable proteins are compact, modular, and can be directed to modify DNA in human cells. Online February 27, 2025.
In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.
By Count Me In Communications March 11, 2025 Count Me In , a patient-partnered research initiative led by the Broad Institute of MIT and Harvard and Dana-Farber Cancer Institute, is launching a new project focused on translocation renal cell carcinoma (tRCC), a rare and aggressive form of kidney cancer.
Why brain cancer is often resistant to immunotherapy By Allessandra DiCorato February 26, 2025 Breadcrumb Home Why brain cancer is often resistant to immunotherapy Researchers find four coordinated gene expression programs in immune cells from glioma tumors, including two that could lead to immunotherapy resistance. Online February 26, 2025.
1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] 1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] 6 February 2025. 6 February 2025. Retrieved 2 April 2025.
The interest from analysts is driven by near-term readouts from the COMPANION-002 Phase 2/3 Study including ORR in March and median PFS later in 2025. In a Phase 2 study of tovecimig + paclitaxel in second and third line BTC, the ORR was 37.5%, the median PFS = 9.4 months and the median OS = 12.5
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Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial.
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The $150 million in fresh funds will help Taysha keep the lights on well into 2025 and support testing of an experimental gene therapy for Rett syndrome.
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Successful Scale-Up Of An Upstream Viral Vector Process Using An Adherent Platform swheeler Thu, 02/27/2025 - 11:40 Thu, 04/10/2025 - 11:00 Resource Type Webinar Brian Gardell Cell and gene therapies offer hope for previously untreatable diseases, with viral vectors currently favored as the primary gene delivery method.
In this edition, our CEO and co-founder, Ariel Katz, along with Regional VP of Trial Landscape, Ryan Brown, share their top predictions for 2025. Ryan Brown , Regional VP, Trial Landscape Global standards for diversity plans will emerge in 2025. Ariel Katz , CEO & Co-Founder AI will be used to combat rare diseases.
On Demand Start Date Thu, 03/06/2025 - 13:00 End Date/Time Thu, 03/06/2025 - 12:00 Listing Image CardinalHealth_ListingLogo_250x190.png png Listing Introduction Optimize patient access and overcome barriers with a structured approach to launch planning.
Fast-Track Gene Therapy Discovery With Easy Access to NHPs blussier Wed, 01/29/2025 - 15:57 HTML Speed Up Your Gene Therapy Discovery at Our Sacramento Site Do you need quick, hassle-free access to a large pool of nonhuman primates (NHPs) for your gene therapy discovery work?
Join OM1 experts in our upcoming webinar to learn how commercial teams can leverage AI-driven real-world insights and ensure their therapies reach the patients who need them most. On Demand Start Date Tue, 04/08/2025 - 14:00 End Date/Time Tue, 04/08/2025 - 14:00 Listing Image OM1_ListingLogo_250x190.png
Nonviral CRISPR-based knock-in of chimeric antigen receptor transgenes into T cells for cell therapy dwunderlin Fri, 02/14/2025 - 14:52 Thu, 04/03/2025 - 11:00 Resource Type Webinar Krishanu Saha, PhD Max Sellman Join us for an insightful webinar on the latest advancements in cell and gene therapy, focusing on the innovative Cas9-CLIPT technology.
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